DK3360580T3 - METHOD OF GENETIC TREATMENT USING THE AAV-XBP1S / GFP VIRUSE AND ITS USE IN THE PREVENTION AND TREATMENT OF AMYOTROPHICAL LATERAL SCLEROSIS - Google Patents

METHOD OF GENETIC TREATMENT USING THE AAV-XBP1S / GFP VIRUSE AND ITS USE IN THE PREVENTION AND TREATMENT OF AMYOTROPHICAL LATERAL SCLEROSIS Download PDF

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DK3360580T3
DK3360580T3 DK16852956.8T DK16852956T DK3360580T3 DK 3360580 T3 DK3360580 T3 DK 3360580T3 DK 16852956 T DK16852956 T DK 16852956T DK 3360580 T3 DK3360580 T3 DK 3360580T3
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treatment
amyotrophical
xbp1s
viruse
aav
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DK16852956.8T
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Danish (da)
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Flores Claudio Andrés Hetz
Paterakis Vicente Spiro Valenzuela
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Univ Chile
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
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    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0089Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y115/00Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001Superoxide dismutase (1.15.1.1)

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Virology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
DK16852956.8T 2015-10-09 2016-09-30 METHOD OF GENETIC TREATMENT USING THE AAV-XBP1S / GFP VIRUSE AND ITS USE IN THE PREVENTION AND TREATMENT OF AMYOTROPHICAL LATERAL SCLEROSIS DK3360580T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CL2015003024A CL2015003024A1 (en) 2015-10-09 2015-10-09 Method of genetic treatment using aav-xbp1s / gfp virus, and its use in the prevention and treatment of amyotrophic lateral sclerosis.
PCT/CL2016/000056 WO2017059554A1 (en) 2015-10-09 2016-09-30 Method for genetic treatment using the aav-xbp1s/gfp virus and use thereof in the prevention and treatment of amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
DK3360580T3 true DK3360580T3 (en) 2022-02-21

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
DK16852956.8T DK3360580T3 (en) 2015-10-09 2016-09-30 METHOD OF GENETIC TREATMENT USING THE AAV-XBP1S / GFP VIRUSE AND ITS USE IN THE PREVENTION AND TREATMENT OF AMYOTROPHICAL LATERAL SCLEROSIS

Country Status (14)

Country Link
US (1) US10512698B2 (en)
EP (2) EP3360580B1 (en)
AU (1) AU2016336063B2 (en)
CL (1) CL2015003024A1 (en)
CY (1) CY1125031T1 (en)
DK (1) DK3360580T3 (en)
ES (1) ES2907684T3 (en)
HR (1) HRP20220199T1 (en)
HU (1) HUE057861T2 (en)
LT (1) LT3360580T (en)
PL (1) PL3360580T3 (en)
PT (1) PT3360580T (en)
SI (1) SI3360580T1 (en)
WO (1) WO2017059554A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CL2015003242A1 (en) * 2015-11-04 2016-10-14 Univ Chile Aav / upr-plus virus, upr-plus fusion protein, genetic treatment method and its use in the treatment of neurodegenerative diseases, such as parkinson's and huntington diseases, among others.
US20220184175A1 (en) * 2019-02-01 2022-06-16 Universidad De Chile Treatment of aging or age-related disorders using xbp1
CN112143693A (en) * 2019-06-28 2020-12-29 杭州康万达医药科技有限公司 Method for producing virus and harvesting solution composition

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
LT2489733T (en) 2006-06-07 2019-05-27 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
LT3146982T (en) 2006-10-03 2019-11-11 Genzyme Corp Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
US20110142799A1 (en) * 2008-06-23 2011-06-16 President And Fellows Of Harvard College Modulation of neurodegenerative disease by modulating xbp-1 activity
CL2014003590A1 (en) * 2014-12-30 2015-07-10 Univ Chile Aav / xbp1s-ha virus, method of genetic treatment and its use in the optimization and improvement of cognitive, memory and learning abilities.

Also Published As

Publication number Publication date
EP3360580A4 (en) 2019-03-13
HRP20220199T1 (en) 2022-04-29
US10512698B2 (en) 2019-12-24
PT3360580T (en) 2022-02-24
ES2907684T3 (en) 2022-04-26
US20190030188A1 (en) 2019-01-31
AU2016336063A1 (en) 2018-05-24
CY1125031T1 (en) 2023-03-24
EP3360580B1 (en) 2021-12-01
EP4035687A1 (en) 2022-08-03
SI3360580T1 (en) 2022-05-31
AU2016336063B2 (en) 2023-07-13
PL3360580T3 (en) 2022-05-23
EP3360580A1 (en) 2018-08-15
WO2017059554A1 (en) 2017-04-13
LT3360580T (en) 2022-05-10
HUE057861T2 (en) 2022-06-28
CL2015003024A1 (en) 2016-05-13

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